USD History

06/19/2026

From Kenneth P. Serbin, professor of history: On June 17, uniQure announced a new plan to apply to the FDA to seek approval of its gene therapy for Huntington’s disease – a dramatic shift after the agency had last year blocked the drug despite results showing, for the first time, that HD progression could be slowed. The announcement comes in the wake of the May 12 resignation of Dr. Marty Makary as head of the crisis-ridden FDA, which has clashed with uniQure. STAT considered Makary to be the “worst” leader in 25 years. uniQure reported that the FDA had accepted that the three-year analysis of the drug, AMT-130, which the company has presented as demonstrating efficacy against HD, can serve as the “primary basis” for a drug approval application. uniQure aims to apply in the third quarter of this year. In alignment with the FDA, the company will also conduct a “confirmatory study” to further test the efficacy of AMT-130. This study would not include a sham surgery as a placebo – a requirement introduced in March by the FDA. uniQure can, for a comparator, still use Enroll-HD, previously a major point of contention. “Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” uniQure CEO Matt Kapusta stated. “Today’s announcement from uniQure represents an encouraging and meaningful step forward for the Huntington’s disease community,” said HDSA CEO Amy Gray, adding that, following “regulatory hurdles,” the HD community “united like never before.” Those efforts included a key, bipartisan June 2 congressional townhall briefing, in Washington, D.C. Democratic Rep. Jake Auchincloss and Republican Rep. Morgan Griffith spoke passionately about FDA reform and getting drugs faster to rare disease communities. “This is the best-case scenario for our community,” Lauren Holder, a Help4HD International advocate and, like me, an HD gene carrier, told STAT after the AMT-130 news. “This happened because dedicated patient advocates refused to give up, because this community continued to show up, speak up, and fight, even when it felt like no one was listening. Today, it feels like we were finally heard.” For more about the whirlwind of events leading up to the uniQure announcement and details of the congressional briefing, see my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

05/17/2026

From Kenneth P. Serbin, professor of history: Huntington’s Disease Awareness Month (May) encourages affected families to share their stories about this rare neurological disorder with the wider world. A 2019 blog posting about HD Awareness Month featured a photo of me pointing to HDSA flyers posted on my office door at the University of San Diego (USD), where I teach history and research science and technology studies. As a fulfillment of a long-term goal to advance both awareness-building and deepen my knowledge of HD science, in 2025 I inaugurated a new course, A History of the Brain: Examining Huntington’s Disease. This semester I distributed a flyer containing HD Awareness Month promotional material from HDSA and HDF. Each holding a flyer, three students – Ana-Lucia Moreno, Ava Puorro, and Mia Wilde – had a picture of me taken with them in the classroom and posted it on Wilde’s Instagram with the title “National Huntington’s Disease Awareness Month.” “Best class ever with Dr. Serbin, who has Huntington’s disease and taught us so much about it in class!” they wrote on the posting. “Watch his blog to learn more about HD and how we can make all people feel included.” I lecture on the basic scientific understanding of the brain from antiquity to the present. To launch discussion about the disease, students do short writing assignments based on the course readings, including my blog. Many days in the course produce deeply emotional debates and discussions. My students’ recognition of the need for social inclusion for all echoes the course’s deep exploration of the stigma and discrimination associated with HD, other neurological disorders, mental illness, and disabilities. The course studies in details HD’s triad of devastating symptoms: involuntary movements, cognitive loss, and behavioral and psychiatric difficulties. We also delve into many other difficult challenges faced by the HD community, such as genetic testing, family planning, and bioethical issues like abortion and su***de. We also discuss the quest for treatments of this still incurable disorder. The course has underscored for me the fact that Huntington’s disease is still not a household word. For most of the students it is their first exposure to HD. One person told Wilde that “it was such a thoughtful thing that we were doing because they had a friend who had Huntington’s disease before, which I thought was very meaningful to hear.” Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

04/23/2026

From Kenneth P. Serbin, professor of history: As the reportedly dysfunctional FDA faces demands for greater clarity in the wake of rare-disease drug denials, uniQure continues to seek a path to get AMT-130, its historically efficacious Huntington’s disease gene therapy, approved. After reneging on its promise to allow uniQure to apply for AMT-130 approval in 2026, the FDA recommended a new, Phase III clinical trial. Sen. Ron Johnson, a Republican, has launched an investigation of the FDA, and a rare disease coalition has urged President Trump to restore regulatory clarity at the agency. A possible avenue to AMT-130 approval opened on April 14. Dr. Teresa Buracchio of the FDA said at the National Organization for Rare Disorders (NORD) symposium that the agency’s “plausible mechanism framework” for approval of bespoke gene therapies might be applied “to approve other therapies.” A bespoke therapy is given to a single individual, such as “Baby KJ,” the world’s first individual to be treated, and successfully, with a personalized (customized) CRISPR gene editing therapy. Dr. Buracchio stated that the FDA is open to applying the framework “conceptually” to Huntington’s disease broadly. A therapy needs to show “substantial evidence of effectiveness and a substantial improvement that’s clear and distinct from the natural history of the disease,” she said, noting that KJ demonstrated marked improvement in symptoms and reached developmental milestones. By comparison, a slowly progressive neurodegenerative disease like HD “is going to be a harder case to make,” Dr. Buracchio said. At the NORD symposium, uniQure’s Dr. David Margolin addressed the FDA’s concerns. Dr. Margolin stated that HD’s slow progression makes it nearly impossible to show clear efficacy over a short period. AMT-130 demonstrated a 75 percent slowing of HD progression over three years. Regarding the use of Enroll-HD patient registry data in place of a placebo in uniQure’s analyses, Dr. Margolin pointed out that the difference between AMT-130 clinical trial participants and the individuals uniQure selected from Enroll-HD was negligible. “I know there’s active dialogue with FDA and the Huntington’s disease organizations regarding how to interpret and best utilize these clinical scored measures, and that’s an ongoing process,” Dr. Margolin stated. In the current political and business climate, perhaps the HD community also needs a celebrity connection, as biotech observers have noted with dark humor. “I guess people living and dying with Huntington’s disease need an influencer/podcaster to text Trump,” said STAT senior biotech writer Adam Feuerstein, who shared an item about podcaster Joe Rogan receiving a promise from Trump of immediate FDA approval for a psychedelic treatment. “That’s how the FDA works these days.” Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

04/08/2026

From Kenneth P. Serbin, professor of history: Two key political developments could aid uniQure’s effort to seek approval of its historically efficacious Huntington’s disease gene therapy, AMT-130, from the FDA: a senatorial probe of the FDA and a letter to President Trump from rare-disease advocates. uniQure is preparing for yet another meeting with the FDA to discuss the potential Phase III large-scale clinical trial required by the agency to further test the efficacy of AMT-130. In September 2025, uniQure revealed that 17 of the individuals receiving the highest dose of AMT-130 in its Phase I/II trial had a slowing of progression of HD by 75 percent. The FDA, seen by many biotech observers to have become dysfunctional under the Trump administration, then surprisingly reneged on a promise to allow uniQure to apply for approval in 2026. On March 9, Wisconsin Senator Ron Johnson, a Republican, announced that he had launched an investigation of the FDA because it had rejected drug applications for several rare diseases. Johnson described the FDA’s request for a new AMT-130 trial, which would include a deeply invasive sham surgery for participants not getting the actual drug, as “bureaucratic idiocy.” The senator added: “We’re going to find out exactly what issues the FDA listed for their ‘nos’.” On April 1 the Rare Disease Advocacy, Biotechnology, and Investor Coalition sent a letter to President Trump and other administration leaders. The letter urged them to restore regulatory clarity at the FDA as it considers a new leader for the agency’s Center for Biologics Evaluation and Research, which evaluates gene therapies such as AMT-130. Its controversial director, Vinay Prasad, M.D., has resigned for the second time after criticism from biopharma executives, investors, and members of Congress. The coalition includes nearly 100 patient disease advocacy groups, biotech executives, and investors. The letter noted that the Center had become less flexible in overseeing rare disease clinical trials. “We believe it is of the utmost importance that the FDA chooses [for the Center] a leader who understands the unique challenges of rare disease development and respects and values the views of patients and physicians,” their letter stated. Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

03/05/2026

From Kenneth P. Serbin, professor of history: Delaying but not blocking AMT-130, uniQure’s emerging gene therapy to slow Huntington’s disease progression, the FDA “strongly recommended” that the company conduct a full-blown, Phase III clinical trial, rather than assess an application for drug approval based on an earlier trial that showed historic efficacy. The news came in a March 2 uniQure press release, relying on official minutes from the company’s January 30 high-priority meeting with the FDA about the AMT-130 program. The drug has been shown to slow the progression of by HD symptoms by as much as 75 percent. This action reflects the more conservative trend of at least parts of the FDA under the Trump administration. “While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue” with the FDA regarding “regulatory flexibility,” uniQure CEO Matt Kapusta stated. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130. We are deeply grateful for the resilience and support of the Huntington’s disease community and remain committed to standing with patients and their families.” On February 24, a top uniQure scientist presented an updated analysis of AMT-130 at the 21st Annual HD Therapeutics Conference, sponsored by CHDI Foundation. Like other potential remedies, AMT-130 lowers (reduces) the amount of defective huntingtin protein. “We have multiple shots on goal in the huntington-lowering arena, things like uniQure,” CHDI CSO Dr. Robert Pacifici said. “While it's true that none of them have made it out the other end with a positive ruling in a pivotal Phase III trial, we're at the precipice of the types of signals that will indicate that there is a clinically meaningful benefit by lowering huntingtin.” Dr. Cristina Sampaio, CHDI’s chief medical officer, observed that companies other than uniQure have faced changes in course with the FDA. The changes in the approval process are “problematic,” she said. Some scientists think AMT-130 needs more work. Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

02/17/2026

From Kenneth P. Serbin, professor of history: With 15 million people estimated to lose their medical coverage because of Trump administration policies, America has once again entered a health crossroads. As a Huntington’s disease gene carrier who for many years hid that fact from the very system that was supposed to help me – “an absolutely absurd situation”– I rejoiced when Obamacare guaranteed coverage for those of us with pre-existing conditions. Like many Americans, my family and I have struggled with many other aspects of this supposed “system.” As an HD advocate, I embrace health care as a human right. We need so much more than Obamacare. I support Senator Bernie Sanders’ Medicare for All Act of 2025. Medicare for All would involve a national health insurance system. It would cover items and services to diagnose, treat, or rehabilitate a health condition, including hospitalization and prescription drugs, mental health, dental and vision services, long-term care, and reproductive care. There would be no co-pays. All U.S. residents would be included from birth. Sanders’ criticisms of the current system ring true with people’s frustrations. In his words, America’s great political challenge is to decide whether to continue to focus on profits, or do we create a system where “every man, woman, and child in this country should, in a cost-effective way, be guaranteed quality and equitable health care regardless of their economic status.” Sanders has a detailed plan to generate trillions of dollars to support Medicare for All. Sanders notes “overwhelming support” for Medicare for All in polls. The bill now has 111 co-sponsors in the House of Representatives and 17 in the Senate. “Scientists will be freed to concentrate on developing breakthrough drugs, rather than tailoring their research so that pharmaceutical firms can maintain record profits,” Sanders adds. In the HD community and beyond, let us join hands to advocate for Medicare for All. Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

01/30/2026

From Kenneth P. Serbin, professor of history: On January 22 Huntington’s disease organizations delivered two petitions to the FDA, demanding that the agency reverse its rollback on uniQure’s application for its HD gene therapy. In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, reflecting what critics saw as growing dysfunction under the Trump administration, the FDA abruptly switched gears regarding its agreed-to plan for considering AMT-130. HD advocates quickly organized two online petitions, garnering 48,000-plus signatures. “We can’t delay any longer,” Lauren Holder, a Help4HD International advocate and (like me) an HD gene carrier desperate for a therapy like AMT-130, said in a Facebook video at the FDA. “It’s important that they hear us.” Holder said that she often hears from HD-affected individuals that “‘I don’t want to be dying from HD; I want to be living with HD.’ These gene therapies and gene-modifying drugs are what get us to that point.” In a final, intense push for signatures, Holder on her January 16 podcast interviewed petition organizer Jeremy Renz (pictured here), whose wife tested positive for HD. “There’s more young people that are testing,” Renz told Holder. “There’s more young people who are at risk that are getting ramped up and ready to fight the fight.” Crucially, the high priority meeting that uniQure will soon hold with the FDA will include a representative of the community. Holder and Renz saluted how the HD community had united to work hard on AMT-130 advocacy. Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

11/19/2025

From Kenneth P. Serbin, professor of history: As uniQure seeks to overcome a decision by the FDA to roll back its consultations regarding the firm’s promising gene therapy for Huntington’s disease, the community has begun to rally by organizing two petitions asking the agency to support the remedy. uniQure and others in the biotech sector believe that the FDA has become dysfunctional under the Trump administration. On November 13, STAT reported on a private dinner held by uniQure CEO Matt Kapusta with investors on November 11. According to the report, uniQure hopes to find a way forward for AMT-130. “We remain fully committed to people living with HD, who have no disease-modifying treatment options,” Tom Malone, uniQure’s senior director of communications, e-mailed me. “We are wholly focused on working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.” The STAT report noted that Kapusta “doesn’t like all the drama” inside the FDA surrounding its upending of uniQure’s plans. Aiming to stabilize the agency, the FDA has named Richard Pazdur, M.D., a 26-year veteran of the entity, to run its center for regulating and approving new drugs. Dr. Pazdur’s appointment is a sign the Trump administration is seriously addressing the “FDA dysfunction,” Kapusta said at the investor dinner. According to STAT, Kapusta’s remarks at the dinner “reflected biotech’s frustration with volatility” at the FDA. uniQure was disturbed by the fact that the FDA’s new message on AMT-130 was “delivered by lower-level staffers” and not senior decision-makers, STAT reported. The firm “feels like it was screwed over by the FDA, and rightfully so,” one investor told STAT. As of November 15, the two online petitions to the FDA from HD advocates have already garnered almost 9,000 signatures. These moving, persuasive petitions effectively portray the devastating impact of HD on patients and families and the historic breakthrough towards a treatment achieved with AMT-130. They effectively demonstrate the profound need for the drug and urgent action by the FDA. I immediately signed one. I urge everyone to do so. Read more in my latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

11/08/2025

From Kenneth P. Serbin, professor of history, Less than six weeks after uniQure announced that its gene therapy drug slowed the progression of Huntington’s disease by 75 percent, the FDA has backtracked on its conversations with the company regarding the timeline and data needed for potential approval of the remedy. The company’s plan to apply for approval of the drug, AMT-130, in early 2026 and launch it into the market later that year may no longer be possible. In a November 3 press release uniQure announced that, after a recent meeting with the FDA, the company believes that the “FDA currently no longer agrees” that data from its clinical trial of AMT-130 using an “external control” as a comparison “may be adequate to provide the primary evidence in support of” an application for approval. The external control refers to data taken from Enroll-HD, the global HD patient registry, as a baseline, rather than those taking a placebo, to compare with those on AMT-130. uniQure had gotten FDA permission to use Enroll-HD. “This is a key shift from prior communications with the FDA in multiple … meetings over the past year,” the uniQure release stated. “Consequently, the timing of the BLA [Biologics License Application] submission for AMT-130 is now unclear.” “We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024,” said uniQure CEO Matt Kapusta. “This news is unexpected, and we are truly disappointed for people living with HD.” At the top of its website’s homepage the FDA has the phrase “first six months of FDA reforms,” including “unleashing cell and gene therapies.” Science and medicine do not act in a vacuum and are impacted by politics and society, as HD family member and historian Alice Wexler has brilliantly illustrated in her writings. The New York Times has reported on the “collapse of the FDA” under Robert F. Kennedy Jr. STAT reported that since uniQure and the FDA had set drug submission “benchmarks” last year, “the agency has undergone considerable changes. Vinay Prasad, a physician with a reputation for taking a more conservative view of gene therapies, now oversees the division with authority over AMT-130.” STAT also reported on the “slow-boiling feud” at the division, which has lost hundreds of employees. Dr. Prasad pushed out “top cell and gene therapy regulators.” On November 5 HDSA and four other HD entities issued a joint statement: following the regulatory update about uniQure, “it’s clear that stronger alignment and collaboration among HD patient and family organizations are more critical now than ever.” Read more in latest article.

HD is a genetically caused brain disorder that causes uncontrollable bodily movements and robs people's ability to walk, talk, eat, and think. The final result is a slow, ugly death. Children of parents with HD have a 50-50 chance of inheriting the disease. There is no cure or treatment.

10/21/2025

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